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Targeting developmental pathways in children with cancer: what price success?

Lia Gore1, James DeGregori, Christopher C Porter

  • 1Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, CO 80045, USA. lia.gore@ucdenver.edu

The Lancet. Oncology
|February 2, 2013
PubMed

Insights

Targeted cancer therapies offer new treatment avenues but present unique side effects in children. Further research is crucial to understand and manage these developmental impacts in pediatric oncology.

Area of Science:

  • Oncology
  • Pharmacology
  • Developmental Biology

Background:

  • Cancer research increasingly focuses on targeted therapeutics that exploit cancer's molecular vulnerabilities.
  • Targeted agents possess distinct side-effect profiles compared to traditional chemotherapy.
  • Pediatric oncologists are accumulating experience with targeted agents, despite limited regulatory approvals for children.

Purpose of the Study:

  • To review the rationale behind targeted therapy in cancer treatment.
  • To discuss challenges in developing targeted drugs for pediatric use.
  • To summarize the unique side-effect profiles and developmental impacts of targeted agents in children.

Main Methods:

  • Literature review of targeted therapy in cancer research.
  • Analysis of challenges in pediatric drug development.
  • Synthesis of data on targeted agent side effects in children.

Main Results:

  • Targeted therapies have shown success but have unique side-effect profiles.
  • Limited data exists on the effects of targeted agents in children.
  • Potential short-term and long-term effects on normal tissue development in children are a concern.

Conclusions:

  • Targeted therapies require careful assessment of developmental effects in pediatric clinical trials.
  • Informed supportive-care strategies are needed for children receiving targeted agents.
  • Future research should prioritize understanding and mitigating unique pediatric side effects.

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